2018
In utero nanoparticle delivery for site-specific genome editing
Ricciardi AS, Bahal R, Farrelly JS, Quijano E, Bianchi AH, Luks VL, Putman R, López-Giráldez F, Coşkun S, Song E, Liu Y, Hsieh WC, Ly DH, Stitelman DH, Glazer PM, Saltzman WM. In utero nanoparticle delivery for site-specific genome editing. Nature Communications 2018, 9: 2481. PMID: 29946143, PMCID: PMC6018676, DOI: 10.1038/s41467-018-04894-2.Peer-Reviewed Original ResearchConceptsSite-specific genome editingReversal of splenomegalyPeptide nucleic acidIntra-amniotic administrationBlood hemoglobin levelsMonogenic disordersNanoparticle deliveryPolymeric nanoparticlesPostnatal elevationGestational ageHemoglobin levelsImproved survivalPediatric morbidityDisease improvementHuman β-thalassemiaReticulocyte countNormal organ developmentMouse modelNormal rangeEarly interventionGenome editingOff-target mutationsPostnatal growthGene editingVersatile method
2001
Intracellular generation of single-stranded DNA for chromosomal triplex formation and induced recombination
Datta H, Glazer P. Intracellular generation of single-stranded DNA for chromosomal triplex formation and induced recombination. Nucleic Acids Research 2001, 29: 5140-5147. PMID: 11812847, PMCID: PMC97609, DOI: 10.1093/nar/29.24.5140.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsBase SequenceCell LineDNADNA, Single-StrandedGenetic VectorsMolecular Sequence DataNucleic Acid ConformationRecombination, GeneticConceptsNovel vector systemMouse cellsInduced recombinationPrimer extension analysisVector systemGenome modificationTriplex formationExtension analysisIntrachromosomal recombinationChromosomal eventsGene expressionSequence insertReporter substrateSuch oligodeoxyribonucleotidesTarget siteSsDNA moleculesIntracellular generationDNARecombinationEfficient intracellular deliveryCellsSuch moleculesSequenceIntracellular deliveryOligodeoxyribonucleotides